Sutimlimab Under Review for Hemolysis in Cold Agglutinin Disease

The Food and Drug Administration (FDA) has granted Priority Review for the Biologics License Application (BLA) for sutimlimab (Sanofi) for the treatment of hemolysis in adult patients with cold agglutinin disease (CAD), a rare type of autoimmune hemolytic anemia.

Sutimlimab is an investigational monoclonal antibody designed to stop hemolysis by selectively inhibiting C1s in the classical complement pathway. The application is supported by data from part A of the open-label, single-arm pivotal phase 3 CARDINAL study that evaluated the efficacy and safety of sutimlimab in 24 patients with primary CAD who had a recent blood transfusion. Patients received an intravenous infusion of sutimlimab through week 26 (Part A) and continued to receive treatment for up to 1 year (Part B).

The primary end point was the proportion of patients with a response, defined by an increase in hemoglobin (Hgb) ≥2g/dL from baseline or reaching a Hgb level ≥12g/dL at the 26-week treatment assessment timepoint, as well as the absence of blood transfusions from weeks 5 to 26 or any other CAD-related treatments.

Results demonstrated that the study met its primary end point with 54% (n=13) of patients achieving the composite end point, with 62.5% (n=15) of patients achieving an increase in Hgb ≥2g/dL or reaching a Hgb ≥12g/dL, and 71% (n=17) of patients being transfusion-free after week 5. Additionally, patients treated with sutimlimab were found to have improvements in Hgb, total bilirubin, and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, key secondary end point of the trial.

“Results from our 26-week pivotal phase 3 study clearly demonstrated that sutimlimab had a clinically meaningful effect on complement-mediated hemolysis, which is the cause of anemia and fatigue,” said John Reed, MD, PhD, Global Head of Research and Development at Sanofi. “If approved, sutimlimab will be the first and only FDA-approved treatment to uniquely address C1-activated hemolysis and help alleviate the chronic disease burden for people with CAD.”

A Prescription Drug User Fee Act (PDUFA) target date of November 13, 2020 has been set for this application. Sutimlimab was previously granted Breakthrough Therapy and Orphan Drug designations by the FDA for this indication.